Multiple imputation of cognitive performance as a repeatedly measured outcome von Rawlings, Andreea Monica, Sang, Yingying, Sharrett, Albert Richey, Coresh, Josef, Griswold, Michael, Kucharska-Newton, Anna Maria, Palta, Priya, Wruck, Lisa Miller, Gross, Alden Lawrence, Deal, Jennifer Anne, Power, Melinda Carolyn, Bandeen-Roche, Karen Jean

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Continued improvement in pulmonary, visceral, biomarker and growth outcomes in children with chronic acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement... von Diaz, George A., Giugliani, Roberto, Guffon, Nathalie, Mengel, Eugen, Scarpa, Maurizio, Witters, Peter, Yarramaneni, Abhimanyu, Li, Jing, Rawlings, Andreea M., Kim, Yong, Ortemann-Renon, Catherine, Kumar, Monica

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Continued improvement in pulmonary outcomes in 3 clinical trials of olipudase alfa in children and adults with chronic acid sphingomyelinase deficiency treated for 2 to 6.5 years von Scarpa, Maurizio, Diaz, George A., Giugliani, Roberto, Lachmann, Robin, Wasserstein, Melissa P., Berger, Kenneth I., Bonella, Francesco, Walsh, Simon L.F., Wuyts, Wim A., Jessel, Andreas, Rawlings, Andreea M., Kumar, Monica

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eP280: Continued improvement in adults with acid sphingomyelinase deficiency after 2 years of olipudase alfa in the ASCEND placebo-controlled trial von Wasserstein, Melissa, Barbato, Antonio, Gallagher, Renata, Giugliani, Roberto, Guelbert, Norberto, Hennermann, Julia, Hollak, Carla, Ikezoe, Takayuki, Lachmann, Robin, Lidove, Olivier, Mabe, Paulina, Mengel, Eugen, Scarpa, Maurizio, Senates, Ebubekir, Tchan, Michel, Villarrubia, Jesus, Thurberg, Beth, Yarramaneni, Abhimanyu, Rawlings, Andreea, Kim, Yong, Kumar, Monica

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Two-year results of the ASCEND trial of olipudase alfa adults with chronic acid sphingomyelinase deficiency show parallel improvements in former placebo patients and further improv... von Wasserstein, Melissa P., Barbato, Antonio, Gallagher, Renata C., Giugliani, Roberto, Guelbert, Norberto B., Hennermann, Julia B., Hollak, Carla, Ikezoe, Takayuki, Lachmann, Robin, Lidove, Olivier, Mabe, Paulina, Mengel, Eugen, Scarpa, Maurizio, Senates, Ebubekir, Tchan, Michel, Villarrubia, Jesus, Thurberg, Beth L., Yarramaneni, Abhimanyu, Rawlings, Andreea M., Kim, Yong, Kumar, Monica

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Continued improvement in pulmonary, visceral, biomarker and growth outcomes in children with chronic acid sphingomyelinase deficiency treated with olipudase alfa enzyme replacement... von Diaz, George A, Giugliani, Roberto, Guffon, Nathalie, Mengel, Eugen, Scarpa, Maurizio, Witters, Peter, Yarramaneni, Abhimanyu, Li, Jing, Rawlings, Andreea M, Kim, Yong, Ortemann-Renon, Catherine, Kumar, Monica

Volltext

Continued improvement in pulmonary outcomes in 3 clinical trials of olipudase alfa in children and adults with chronic acid sphingomyelinase deficiency treated for 2 to 6.5 years von Scarpa, Maurizio, Diaz, George A, Giugliani, Roberto, Lachmann, Robin, Wasserstein, Melissa P, Berger, Kenneth I, Bonella, Francesco, Walsh, Simon L.F, Wuyts, Wim A, Jessel, Andreas, Rawlings, Andreea M, Kumar, Monica

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